Efficacy of long-term treatment with thalidomide in children and young adults with Crohn disease: Preliminary results

Sergio Facchini, Manila Candusso, Stefano Martelossi, Mario Liubich, Elisabetta Panfili, Alessandro Ventura

Research output: Contribution to journalArticle


Background: Several proinflammatory cytokines are involved in the pathogenesis of inflammatory bowel diseases. A significant role has been given to tumor necrosis factor α (TNF-α) as a guide proinflammatory cytokine. Thalidomide selectively reduces TNF-α production by inflammatory cells. The aim of the study was to assess the efficacy of thalidomide to induce and maintain remission in refractory Crohn disease. Methods: The decision to administer thalidomide was made on the basis of patient intolerance or resistance to conventional medical treatment or as the last medical resort before surgical intervention. Only 5 of 96 patients with inflammatory bowel disease satisfied these criteria. All five patients had Crohn disease (male; mean age, 17 years). Thalidomide was administered at night at a dose of 1.5-2 mg/kg/day. The Pediatric Crohn Disease Activity Index, modified Harvey-Bradshaw scores, and steroids reduction were used to assess clinical response, Results: Disease activity decreased consistently in four patients with a reduction of mean Pediatric Crohn Disease Activity Index from 36,9 to 2,5 and the mean Harvey-Bradshaw from 8.5 to 0.75 after 3 months of treatment. Steroid treatment (mean dose, 35 mg/day before treatment) was tapered and then discontinued, in four patients, within 1-3 months. Four patients are in remission after 19-24 months of treatment. The fifth patient discontinued thalidomide after 1 week because of distal paresthesia. Conclusion: Thalidomide seems to be an effective and safe treatment in patients with refractory Crohn disease. This is the first report of long-term use of thalidomide in refractory Crohn disease in pediatric patients.

Original languageEnglish
Pages (from-to)178-181
Number of pages4
JournalJournal of Pediatric Gastroenterology and Nutrition
Issue number2
Publication statusPublished - 2001


  • Inflammatory bowel disease
  • Thalidomide
  • TNF-α

ASJC Scopus subject areas

  • Gastroenterology
  • Histology
  • Medicine (miscellaneous)
  • Food Science
  • Pediatrics, Perinatology, and Child Health

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